With more than two million page views and more than 4,500 items, this blog provides news and commentary on public policy, business and economic issues related to the $3 billion California stem cell agency, officially known as the California Institute for Regenerative Medicine(CIRM). David Jensen, a retired California newsman, has published this blog since January 2005. His email address is djensen@californiastemcellreport.com.

Thursday, November 30, 2017

The California stem cell agency today handed out $16.4 million in research grants seeking therapies for afflictions ranging from gum disease and cancer to vision loss and Parkinson's Disease.

The award for Parkinson's was relatively tiny -- only $150,000 -- but represented a rare case in which the agency's governing board overturned its reviewers, who make the de facto decisions on awards.

The reversal came after one board member, David Higgins, of San Diego, who has Parkinson's, noted that the most common drug that Parkinson's patients take is 70 years old. He told the board.

David Higgins, CIRM photo

“I’m a fourth generation Parkinson’s patient and I’m taking the same medicine that my grandmother took. They work but not for everyone and not for long. People with Parkinson’s need new treatment options and we need them now. That’s why this project is worth supporting. It has the potential to identify some promising candidates that might one day lead to new treatments.”

The largest award today, $5.6 million, went to Anthony Oro of Stanford, who will be testing a therapy to treat an affliction that creates wounds that will not heal. Dan Kaufman of UC San Diego received $5.5 million to produce "killer cells" to help people with a form of leukemia. Catriona Jamieson, also of UC San Diego, received $2.7 million for leukemia research.

Knoepfler is the sole judge of the winner and personally provides a prize of $2,000.

The current voting, which ends Dec. 12, will whittle the list down to 10 from which Knoepfler will pick the winner. Earlier this month, he had this to say about the award,

"Unlike some other science awards out there that shall remain nameless,
the Stem Cell Person of the Year prize is not an insider kind of thing,
but more of an anti-old boy’s club award. It’s not about who you know,
but what you do to help science, medicine, and other people."

Personally, I think the team at the California stem cell agency should be on the list. The folks, past and present, at the agency have labored mightily since 2004 to advance stem cell research. Sometimes the circumstances have been difficult and challenging, but the work continued nonetheless. Maybe next year, as they say in the sports world.
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OAKLAND, Ca. -- Facing the likelihood of a slow and withering death, the California stem cell agency today edged gingerly forward on a path of "cuts" and risky fund-raising in hopes that its research results will soon generate voter support for more billions of dollars.

Two governing board committees of the $3 billion agency, formally known as the California Institute for Regenerative Medicine (CIRM), this afternoon recommended that the full board "entertain" the proposals at its Dec. 14 meeting.

The agency is down to its last $269 million and expects to halt new awards in 2019 unless additional funds are raised between now and the beginning of 2020. It has been running at a $300-million-a-year pace recently and has pumped out about $25,000 an hour, 24 hours a day, seven days a week since it started making awards in 2005, according to calculations by the California Stem Cell Report.

Today's meeting focused on both short and long term finances. The committees examined a proposal to cut its planned clinical awards by $68 million over the next two years by limiting their size. The committees also indicated support for an ambitious effort to raise $222 million privately between now and early 2020.

Longer term, directors staked their hopes on voter approval of a ballot initiative in November 2020 that could total $5 billion. Development of a stem cell therapy that would resonate with voters would be a major key to the success of that effort. The 2004 campaign raised expectations that stem cell cures were right around the corner, but so far the agency has not backed one for widespread use.

Robert Klein, a Palo Alto real estate investment banker who ran the 2004 campaign that created the agency, told the CIRM directors of a private poll that he said showed 70 percent of Californians supported stem cell research and continued funds for the stem cell agency.

Following the meeting, the California Stem Cell Report asked him for a copy of the poll. He declined to provide it or identify the firm that conducted the survey.

Klein told the CIRM board members that the agency has made "remarkable progress" and has created a "moral imperative" to continue the search for stem cell cures. He said,

"The bridge to the future is CIRM and stem cell therapies."

The now less-than-robust finances of CIRM are a product of the ballot initiative, Proposition 71, that created it. CIRM is not funded through the customary process used by most state agencies. The initiative stipulated that CIRM would be supported by $3 billion in state bonds whose funds would flow directly to the agency and bypass the legislature and the governor. When that cash runs out, the agency is left with no other source of state funding.

Raising $222 million privately over the next two years is no small task, but deep pockets exist that may well be tapped. The Eli and Edythe Broad Foundation, for example, has donated nearly $80 million to stem cell research centers at UCLA, UC San Francisco and the University of Southern California. All three are linked to CIRM. In San Diego, the Sanford Stem Cell Consortium was given $100 million in 2013 by Denny Sanford, another billionaire. The consortium came into being after it was backed by $43 million from CIRM.

Nonetheless, competition for philanthropic cash is heated. Thomas and Klein, however, both indicated that they would collaborate on raising the $222 million, which would allow the agency to add eight new clinical awards in 2020, among other things.

The risk lies in the timetable for bringing in the cash. The agency has a relatively tight schedule for making awards --- a schedule that a major philanthropist may or may not be ready or willing to comply with.

Without more cash, the stem cell agency projected that it would wither away by 2023 as it managed a declining number of old research grants.

OAKLAND, Ca. -- A key group of directors of the $3 billion California stem cell agency this afternoon opened a discussion dealing with its short and long-term financial future at 1:04 p.m. The meeting is scheduled to last until 4 p.m. The California Stem Cell Report will file stories as warranted.
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The stem cell agency outlined its assessment of its current position in this slide prepared for today's meeting.

OAKLAND, Ca. -- California's $3 billion stem cell agency is preparing to put the squeeze on its research awards over the next two years, cutting the caps on new grants in some areas by 50 percent or more.

It is all part of a strategy to prolong the life of the 13-year-old research effort as it fights to fulfill the expectations of voters who created it when they approved a ballot initiative in 2004. Voters provided only $3 billion in funding, however, and the agency is now expected to run out of cash for awards in 2019 unless it changes its financial ways.

Meeting here today are two key groups, the Transition and Science subcommittees of the governing board of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known.

They are scheduled to wrestle with both the agency's short-term finances and the longer term effort to extend CIRM's life for perhaps an additional 10 years or more via another ballot initiative in 2020. As for next year, based on past discussions and practices it appears certain that the agency's directors will reduce the size of awards to the scientists and businesses who are often hard-pressed to find non-governmental support.

Phase 1 and phase 1/2 clinical trial grants would see their caps reduced from $20 million to $12 million for nonprofits and $8 million for for-profit enterprises. The average award in 2017 in these categories was $10.3 million.

Phase 2 trials would see their caps cut from also $20 million to $15 million for both non-profit and for-profit enterprises. The 2017 average award was $15.3 million.

Phase 3 trial awards, which averaged $16.7 million in 2017, would have their caps cut from $20 million to $10 million for both types of enterprises.

Millan's presentation indicated that would her proposals would allow the agency to support 24 new trials and eight trial candidates over the next two years, in keeping with a strategy of taking "more shots on goal" as the agency tries to develop a stem cell therapy for widespread use. Clinical trials, which can take years, are the last stage before a therapy is approved by the federal government.

Millan's plan would also allot only $10 million for what the agency calls "Discovery" awards (basic research) next year (10 projects) and $9.25 million in 2019 (nine projects). Discovery awards are expected to hit $46 million this year. Education awards are expected to total $1 million this year. Next year they would receive $750,000 and nothing in 2019.

CIRM did not release details of its strategy for raising the $220 million. CIRM Chairman Jonathan Thomas, however, has already been on the fundraising trail and reports that he has identified a number of potential donors.

The financial plans and any changes will go to the full board for ratification at a meeting Dec. 14 here at CIRM headquarters.

The California Stem Cell Report will cover today's meeting and file reports as warranted on this web site. Below is another presentation slide from the stem cell agency.

Monday, November 20, 2017

Cash is flowing out the door fast at California’s $3 billion stem cell agency, which now expects money to run out for new research awards by the end of 2019 instead of the middle of 2020.

The California Institute for Regenerative Medicine (CIRM), as the agency is formally known, expects to be down to its last $269 million in uncommitted funds by end of this year. And it is working on a new plan to raise $200 million in private funds to sustain its operations through 2020.

The financial projections -- crafted as the agency faces what its leaders call a "critical stage" -- are online this week on the CIRM web site and include proposed lower caps on the size of awards in 2018 and 2019. The agency said the increase this year in the pace of funding was generated by more, higher quality research applications.

Maria Millan, CIRM photo

In documents prepared for a key CIRM meeting next Monday, Maria Millan, CEO of the agency, said her budget scenario would maintain CIRM’s “value proposition as intact as possible” while work moves forward on a proposed, multibillion dollar bond measure in November 2020. She also said enough funds would be available to “wind down” the agency if the bond measure fails.

The agency was created by a ballot initiative in 2004, Proposition 71, which financed it with a $3 billion bond measure. No other significant funding was provided. The ballot campaign raised rosy expectations among voters that stem cell therapies were right around the corner.

So far the agency has not backed a therapy that has been approved for widespread use. But it is helping to support 38 clinical trials, the last stage before a therapy is approved by the federal government for widespread use. Many more trials, which can take years, are expected to be funded prior to 2020,

Thomas’ proposal, in addition to private fundraising, calls for a “citizen-led” bond measure in the fall of 2020 to sustain CIRM on a more long-term basis. During next week's meeting, Robert Klein, the Palo Alto real estate investment banker who led the 2004 ballot initiative campaign, is expected to address the prospects for another initiative measure, possibly as large as $5 billion.

As back-up options, Thomas identified the possibility of asking the legislature and governor to place a bond measure on the 2020 ballot along with seeking donor funds for co-funding of specific projects.

In Millan's presentation, she said it is “essential to preserve CIRM’s value proposition to increase the probability of and the speed by which stem cell treatments can reach patients.”

The session on Monday involves the Transition and Science subcommittees of the CIRM board. The committees are expected to recommend budget and fundraising plans to be ratified by the full board at its meeting Dec. 14.

Next week's meeting will be based at CIRM’s headquarters in Oakland with telephonic sites where the public can participate in La Jolla, Duarte, South San Francisco, Los Angeles and San Francisco. Public comments can be emailed to mbonneville@cirm.ca.gov. The session will also be audiocast on a listen-only basis. Instructions can be found on the agenda along with addresses for the telephonic locations.

Sunday, November 19, 2017

In the Parkinson’s therapy item on Nov. 17, 2017, the California Stem Cell Report incorrectly described the amount of funding involved in the GForce Parkinson’s initiative. The backing includes $52.3 million plus substantial support from BlueRock Therapeutics, which is financed with $225 million from Bayer AG and Versant Ventures. BlueRock, a Cambridge, Mass., firm, says on its web site, “Our most advanced therapeutic candidate, for Parkinson’s disease, will enter the clinic in 2018.”Sphere: Related Content

Jeanne Loring, director of the Scripps Center for Regenerative Medicine in La Jolla, Ca., and also a participant in the GForce initiative, said this week that CIRM has not supported Parkinson's research at the level of the other enterprises involved in GForce.

In an item she wrote for The Niche, a blog published by UC Davis researcher Paul Knoepfler, she listed $52.3 million in support plus substantial backing from BlueRock Therapeutics, which is financed with $225 million from Bayer AG and Versant Ventures. BlueRock, a Cambridge, Mass., firm, says on its web site, “Our most advanced therapeutic candidate, for Parkinson’s disease, will enter the clinic in 2018.

Parkinson's is a devastating disease that afflicts 10 million people in the world and 125,000 in California. One of those persons, David Higgins of San Diego, currently serves on the board of the stem cell agency, formally known as the California Institute for Regenerative Medicine (CIRM). Another, Joan Samuelson, was one of the original board members in 2004 and a fervent but often frustrated voice for Parkinson's research at many CIRM board meetings.

Actor Michael J. Fox, who made TV ads for the 2004 ballot initiative that created the stem cell agency, is also among the those living with the disease. Others with the affliction included the late boxing champion Muhammad Ali and famed semiconductor pioneer Andy Grove.

Loring wrote on The Niche about a meeting in Japan earlier this year dealing with the research teams in the GForce project. She said,

"The Kyoto meeting was unprecedented in my experience. Instead of
competing, the four groups cooperated and shared plans for their
proposed clinical trials. We agreed to harmonize our trials and stay in
communication about our progress. All of us plan to start clinical
trials within two years."

Loring continued,

"Since my team has been recognized by the international GForce
initiative devoted to safe effective therapy for PD, we hope that CIRM
will follow the example of New York, the EU, and Japan, and invest more
in our project to provide neuron replacement therapy for Californians
with Parkinson’s disease.

"While we hope to gain more support from CIRM, we are determined to
follow through with our clinical trial, with or without CIRM. It will
just be more difficult without their help. The patients and their
advocates inspire us, and we won’t let them down."

"We started our pre-clinical PD studies in 2011, with funding from Summit for Stem Cell. The first and only funding we received from CIRM for PD research was in 2016.

"Before 2011, CIRM invested $41,838,336. Since 2011, CIRM has invested $7,357,468. This means that the majority of the funding went to projects that didn’t lead to any translational or clinical applications.

"There are currently three active grants working on Parkinson’s disease, for a total of $4.9 million. We have the only translational grant, and it will expire in March 2018. All but $650,000 runs out by the spring of 2018.

"There are no more grants forthcoming for our work. The Scaled Biolabs grant awarded this year is a partnership with us. Birgitt Schuele’s grant is basic research, not a cell therapy. (Below are the three active grants identified by Loring.)

The stem cell agency, known formally as the California Institute for Regenerative Medicine (CIRM), projects its cash for new awards will run out in about 2 1/2 years. At that point, unless more money is forthcoming, CIRM will only be overseeing the dwindling number of awards whose terms extend beyond June of 2020.

The agency's fate was dictated by Proposition 71, which created CIRM in 2004. It also provided $3 billion in state bond funding, which flows directly to CIRM without oversight by the governor or the legislature. No additional, significant resources were contained in the ballot initiative.

CIRM's future has been an occasional topic for its board for some time. But the issue has taken on more urgency this year. At a meeting in September of a newly formed Transition Subcommittee of the governing board, CIRM Chairman Jonathan Thomas said in what may have been an understatement,

"CIRM, as we know, is at a critical stage of its mission here."

The meeting on Nov. 27 will additionally involve the board's Science Subcommittee. What emerges from the session will go to the full, 29-member board in December for ratification.

Options in September included a multi-billion dollar bond ballot measure in 2020 and a possible merger, which was described as something of a last resort. Whatever path is chosen, it likely would lead to changes in the agency, which has been criticized for conflict of interest issues and its dual executive arrangement, among other matters.

The agency has awarded $2.3 billion in 919 grants during its 13-year history. About 90 percent of the awards has gone to institutions with links to members of the governing board, past and present, according to calculations by the California Stem Cell Report.

So far the agency has not fulfilled expectations of voters that it would generate a widely available therapy. Something may emerge in the next couple of years from the 38 clinical trials currently backed by the agency. (Forty-three have been funded but five were terminated.) The trials, which can take years, are the last stage before a therapy is approved by the Food and Drug Administration for widespread use. CIRM plans to add more trials in the next couple of years.

This month's meeting will be based at CIRM's Oakland headquarters with teleconference locations elsewhere in the state where the public can take part. It is scheduled to begin at 1 p.m. PST and run until 4 p.m.

The California Stem Cell Report will provide full coverage of the meeting that day with advance information as it is posted on the CIRM web site.

Sunday, November 05, 2017

An Asterias video carried by KQED as part of its story on a CIRM-backed stem cell therapy.

Just before Halloween, the $3 billion California stem cell agency chalked up another PR score with a long, favorable piece on a public television station that covers the San Francisco Bay area.

The story focused on a potential therapy for spinal cord injury developed by Asterias Therapeutics, Inc., of Menlo Park, Ca. The audience for the story was also critically important -- millions of voters who may well be asked to provide more billions for the stem cell agency, which is slated to run out of cash in mid 2020.

The piece by David Gorn carried caveats, but it also used quotations such as "incredibly exciting" and phrases such as "truly remarkable." And it noted -- relatively high in the story -- that the research is backed by the California Institute for Regenerative Medicine (CIRM), as the stem cell agency is formally known.

Gorn wrote,

"The trial is legitimate. It’s partially funded by the California Institute for Regenerative Medicine, the state’s stem cell agency; well-known spinal experts are participating; and the FDA has certified the treatment as one that preliminary clinical evidence indicates has the 'potential to address unmet medical needs' related to a 'serious or life-threatening disease or condition.'"

The good news about the Asterias therapy has been written about before. But it is far from a topic that is talked about at California breakfast tables.

The agency's activities -- good, bad or indifferent -- are well out of the coverage of the mainstream media, a sharp change from its early days in 2005 and 2006. Raising its profile -- favorably -- is a daunting task given that science writers have virtually vanished from the mainstream media -- all part of the shrinking world of today's journalism.

In the last couple of years, however, the agency has stepped up its funding of clinical trials, which are the last stage before a therapy is approved for widespread use. Although clinical trials can take years to complete, their initial results can resonate with the public in a way that basic research does not. Seeing a person overcome an affliction is far more compelling than watching a mouse recuperate.

CIRM's efforts are additionally hampered by a convention in science journalism that tends to minimize the importance of sources of funding. In most cases, it is mentioned only at the end of articles. Sometimes it is omitted entirely.

CIRM, however, is a grand, California experiment that took up the cause of stem cell research when it was suffering from a lack of attention from risk averse companies and a lack of support from the federal government. Many of the 921 projects that the agency has funded may have never gotten off the ground without support from the voters of California, who created the agency in 2004 through a ballot initiative.

CIRM additionally carries policy implications that go beyond bench science. It is the first such effort in state history, marrying big science, big academia, big business and big politics in a unique way in California.

If the agency is to continue financing research to the tune of $300 million a year, it is almost certain to need another ballot measure. And to win voter approval once again, CIRM will need a vault filled with stories of accomplishment and human emotion that will resonate with the voters of the Golden State.

(Editor's note: The next to last paragraph is slightly rewritten from an earlier version of this item.)

The piece contains no big surprises but is worth examining if you want to get a sense of her background and where she is headed. Here are two excerpts.

Question: "What will happen once CIRM has given out all $3 billion allocated to it by Proposition 71?"

Answer: "We are working with our board to pursue sustainability options to ensure that the products of the Proposition 71 investment have the best chance of reaching the patients. CIRM has played an essential role in growing the stem cell/regenerative medicine field by bringing in the critical mass of resources, top notch researchers, rigorous basic and translational research and building the most robust late development and clinical stage portfolio to accelerate novel treatments to patients with unmet medical needs. With the CIRM 2.0 processes that drove operational excellence and acceleration, there is incredible potential to build upon the recent early successes that led to projects that are nearing FDA marketing approval and to drive more of these successes to the benefit of healthcare and patients."

Question: "What are your plans for CIRM over the next few years?"

Answer: "We are fully committed and motivated to executing on the 5 year strategic plan that we launched in January 2016. When we proposed these strategic goals, they were 'stretch' goals and now it looks like we will achieve these goals. This strategic plan is driven by our mission 'to accelerate stem cell treatments to patients with unmet medical needs.'"

About Me

The California Stem Cell Report is the only nongovernmental website devoted solely to the $3 billion California stem cell agency. The report is published by David Jensen, who worked for 22 years for The Sacramento Bee in a variety of editing positions, including executive business editor and special projects editor. He was the primary editor on the 1992 Pulitzer Prize-winning series, "The Monkey Wars" by Deborah Blum, which dealt with opposition to research on primates. Jensen served as a press aide in the 1974 campaign and first administration of Gov. Jerry Brown. (Time served: two years and one week.) He writes from his sailboat on the west coast of Mexico with occasional visits to land. Jensen began writing about the stem cell agency in 2005, noting that it is an unprecedented effort that uniquely combines big science, big business, big academia, big politics, religion, ethics and morality as well as life and death. The California Stem Cell Report has been identified as one of the best stem cell sites on the Internet. Its readership includes the media (both mainstream and science), a wide range of academic/research institutions globally, the NIH and California policy makers.